FDA Commissioner Resignation is a Sign of the Times

Numerous reports are coming in that FDA Commissioner Margaret Hamburg is stepping down from her position. Her tenure as FDA Commissioner has seen the largest growth in Orphan Drug designated treatments since the inception of the Orphan Drug Act in 1983.

For those who have been following my reporting on Obama’s new precision medicine initiative, this shouldn’t come as a major surprise. Margaret Hamburg has been one of the single largest proponents of Rare Disease and Orphan treatments, and it is no coincidence that under her tenure, one of the longest ever for the role, there was such tremendous growth in that field.

Her decision to step down is indicative of the direction we are headed in both science and regulation. In the six years of her authority, science has changed tremendously. Obama’s precision medicine initiative is the capstone to that change, and Dr. Hamburg’s resignation signifies a passing of the torch.

Dr. Hamburg worked meticulously to get treatments for some of the patients with the rarest diseases. But as we leave the era of “rare” and “common”, and enter the era of “individualized” medicines, a new Commissioner will need to deal with changing realities. It is no longer about disease occurrence, but about genomics. It is no longer about ensuring return on investment for a small population, but for an individual’s medication.

Dr. Hamburg has done years worth of laying the groundwork to allow us to achieve everything we have in the rare disease space. Now, it is time for a new commissioner to do the same with Precision Medicine.

As we say goodbye to Dr. Hamburg in March, we must all acknowledge a harsh reality: we are saying goodbye to the Orphan Drug industry as we know it. And that’s a good thing.